Company lists · Data refreshed September 2026

Top 25 Familial hypercholesterolemia treatment companies worldwide

RevenueBase identified 61 familial hypercholesterolemia treatment companies worldwide as of September 2026 and tracks 8,648 professional contacts across them, including 2,258 with verified work emails. The United States is the largest market and Boston the largest hub.

This list covers familial hypercholesterolemia treatment companies worldwide, matched on what each company actually does as described in its own words. Ranked by team size, largest first. Every row shows the company's own description, its RevenueBase Smart Search rating, and when it was last checked. Data refreshed September 19, 2026. Country and city lists for familial hypercholesterolemia treatment companies follow the table.

Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase Smart Search, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.

How to read this list HQ checked on every row RevenueBase Smart Search rating on every row Refreshed Sep 19, 2026 86% judged on-target in blind review
61Companies
8,648Contacts tracked
2,258Verified emails
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The 25 largest familial hypercholesterolemia treatment companies worldwide

Ranked by team size
Top 25 of 61 familial hypercholesterolemia treatment companies worldwide, ranked by team size, largest first. Data refreshed Sep 19, 2026. RevenueBase Smart Search rates how each company's information relates to familial hypercholesterolemia treatment companies: strong evidence (its own description names it), partial (names part of it), indirect (matched on the rest of its profile rather than the description shown). Click a contact count to view that company's people.
#CompanyRevenueBase Smart SearchHeadquartersEmployeesContacts
1Indirect evidenceSan Rafael, CA, United StatesHQ checked Sep 19, 20261,001-5,0003,943 contacts →

Indirect evidence

“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.”

Source: RevenueBase Database · checked Sep 19, 2026
2Indirect evidenceCarlsbad, CA, United StatesHQ checked Sep 19, 2026501-1,0001,313 contacts →

Indirect evidence

“For three decades, Ionis has invented medicines that bring better futures to people with serious diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
3Indirect evidencePalo Alto, CA, United StatesHQ checked Sep 19, 2026501-1,000837 contacts →

Indirect evidence

“We exist to bring transformative medicines to people living with genetic conditions.”

Source: RevenueBase Database · checked Sep 19, 2026
4Indirect evidenceAnn Arbor, MI, United StatesHQ checked Sep 19, 2026201-500313 contacts →

Indirect evidence

Source: RevenueBase Database · checked Sep 19, 2026
5Indirect evidencePittsburgh, PA, United StatesHQ checked Sep 19, 2026201-500300 contacts →

Indirect evidence

“A commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines for high unmet medical needs.”

Source: RevenueBase Database · checked Sep 19, 2026
6Partial evidenceEmeryville, CA, United StatesHQ checked Sep 19, 202651-200236 contacts →

Partial evidence

“4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented…”

Source: RevenueBase Database · checked Sep 19, 2026
7Indirect evidenceBoston, MA, United StatesHQ checked Sep 19, 2026201-500189 contacts →

Indirect evidence

“Verve Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, is developing a new class of genetic medicines for cardiovascular disease with the potential to transform…”

Source: RevenueBase Database · checked Sep 19, 2026
8Indirect evidenceBoston, MA, United StatesHQ checked Sep 19, 202651-200160 contacts →

Indirect evidence

“Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including…”

Source: RevenueBase Database · checked Sep 19, 2026
9Indirect evidenceSouth San Francisco, CA, United StatesHQ checked Sep 19, 202651-200119 contacts →

Indirect evidence

“Tenaya Therapeutics is a clinical stage biotechnology company.”

Source: RevenueBase Database · checked Sep 19, 2026
10Indirect evidenceAventura, FL, United StatesHQ checked Sep 19, 202651-20088 contacts →

Indirect evidence

“This page is intended for US audiences.”

Source: RevenueBase Database · checked Sep 19, 2026
11Indirect evidenceCambridge, MA, United StatesHQ checked Sep 19, 202651-20084 contacts →

Indirect evidence

“We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…”

Source: RevenueBase Database · checked Sep 19, 2026
12Indirect evidencePalo Alto, CA, United StatesHQ checked Sep 19, 202611-5061 contacts →

Indirect evidence

“GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
13Indirect evidencePhiladelphia, PA, United StatesHQ checked Sep 19, 202651-20056 contacts →

Indirect evidence

“GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living…”

Source: RevenueBase Database · checked Sep 19, 2026
14Indirect evidenceLake Forest, IL, United StatesHQ checked Sep 19, 202651-20053 contacts →

Indirect evidence

“Jaguar Gene Therapy, LLC is dedicated to accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases including those that impact sizeable…”

Source: RevenueBase Database · checked Sep 19, 2026
15Indirect evidenceUnited StatesHQ checked Sep 19, 20261,001-5,00035 contacts →

Indirect evidence

“Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
16Indirect evidenceSan Diego, CA, United StatesHQ checked Sep 19, 202651-20025 contacts →

Indirect evidence

“ViaCyte is now part of Vertex Pharmaceuticals.”

Source: RevenueBase Database · checked Sep 19, 2026
17Partial evidenceAmsterdam, NetherlandsHQ checked Sep 19, 20261-1021 contacts →

Partial evidence

“FH Europe Foundation is an umbrella organisation of international patient organisations, actively working together to secure early identification and diagnosis of Familial…”

Source: RevenueBase Database · checked Sep 19, 2026
18Partial evidenceBedford, MA, United StatesHQ checked Sep 19, 202651-20017 contacts →

Partial evidence

“Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
19Indirect evidenceNovato, CA, United StatesHQ checked Sep 19, 20261-1016 contacts →

Indirect evidence

“Cyclarity Therapeutics, Inc, is pursuing a mission to treat the underlying causes of age-related disease.”

Source: RevenueBase Database · checked Sep 19, 2026
20Indirect evidenceUmeå, SwedenHQ checked Sep 19, 20261-1012 contacts →

Indirect evidence

“We are lipid biology experts focusing on developing novel therapeutics for patients with lipid related disorders.”

Source: RevenueBase Database · checked Sep 19, 2026
21Indirect evidenceSyracuse, NY, United StatesHQ checked Sep 19, 20261-1011 contacts →

Indirect evidence

“Repair Biotechnologies is a preclinical biotechnology company focused on developing drugs for cholesterol and aging-related diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
22Indirect evidenceKing Of Prussia, PA, United StatesHQ checked Sep 19, 202611-5010 contacts →

Indirect evidence

“XyloCor Therapeutics is a development-stage, biotechnology company dedicated to developing first-in-class gene therapies for patients with unmet medical need in cardiovascular…”

Source: RevenueBase Database · checked Sep 19, 2026
23Indirect evidenceMelbourne, AustraliaHQ checked Sep 19, 20261-1010 contacts →

Indirect evidence

“Percheron Therapeutics Limited (ASX: PER) is an international biotechnology company focused on the development of novel therapies for rare diseases.”

Source: RevenueBase Database · checked Sep 19, 2026
24Indirect evidenceUnited StatesHQ checked Sep 19, 20261-108 contacts →

Indirect evidence

“Glafabra is a biotherapeutics development company focused on bringing into the clinic our Live-cel(TM) technology for cell-based gene therapy (Cb-GT).”

Source: RevenueBase Database · checked Sep 19, 2026
25Indirect evidenceCincinnati, OH, United StatesHQ checked Sep 19, 20261-107 contacts →

Indirect evidence

“LIB Therapeutics is a privately held, late-stage biopharmaceutical company dedicated to bringing novel, safe and convenient subcutaneous and oral PCSK9 inhibitors to the millions…”

Source: RevenueBase Database · checked Sep 19, 2026

Showing 25 of 61 familial hypercholesterolemia treatment companies worldwide

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Familial hypercholesterolemia treatment companies worldwide at a glance

As of Sep 19, 2026
26.1%Verified-email coverage
141.8Average contacts per company
11-50Median size (employees)
4.9%Largest-city concentration · Boston
Sep 19, 2026Page data refreshed

Companies by employee count

1-10
28
11-50
13
51-200
11
201-500
4
501-1,000
3
1,001-5,000
2

Email recency is reported on each country list linked below.

How this list was built

Company categories are rarely black and white. RevenueBase reads each company's own description of its business to decide whether it belongs among familial hypercholesterolemia treatment companies, so some rows will be adjacent to the category rather than pure plays. The description and RevenueBase Smart Search rating on every row are there so you can check each match yourself.

Source
The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase Smart Search, the natural-language company search available to every RevenueBase user.
Coverage
Every country. Country and city lists for familial hypercholesterolemia treatment companies are linked below.
What qualifies
Companies worldwide whose own description of what they do matches “familial hypercholesterolemia treatment companies”. Matching reads each company's description, not its industry code or its name.
RevenueBase Smart Search
RevenueBase Smart Search is how this list is assembled and how each row is rated. RevenueBase's data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to a category defined in a user's own words. Each row is rated strong evidence (its own description names familial hypercholesterolemia treatment companies), partial evidence (it names part of the category) or indirect evidence (the description shown names none of it and the match rests on the rest of the company's profile). The description supporting each rating is quoted on the row.
What we exclude
Companies whose own description does not clearly place them among familial hypercholesterolemia treatment companies, and records with no verified work email.
Match quality
In blind human judging of this matching method, 86% of matched companies were on target. Expect roughly 1 in 7 rows to be adjacent to the category rather than a pure play. Every row shows the company's own description and its RevenueBase Smart Search rating so you can judge each match yourself. Read the full methodology: how the lists are built, how the judging worked, and what the figure means.
Ranking
Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
Verification
Every listed company carries at least one verified work email, 2,258 across the full list. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
Refresh
This page's figures were pulled from the company graph on September 19, 2026. Lists refresh periodically as the graph is re-checked.
Corrections
Wrong category, wrong headquarters, or a company that has closed? Use “Report an issue” on any row. Reports go to the RevenueBase Data Team and feed back into the company graph.

Familial hypercholesterolemia treatment companies by location

1 lists · largest first

Every Familial hypercholesterolemia treatment companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.

Countries

Frequently asked

What is RevenueBase Smart Search?

RevenueBase Smart Search is how RevenueBase decides which companies belong on this list and how strongly each one fits. Its data engine and AI assess a company's LinkedIn profile, information supplied directly by the company, its website and other publicly available information, then relate it to the category the list is about. Each row is rated strong evidence when the company's own description names familial hypercholesterolemia treatment companies, partial evidence when it names part of the category, and indirect evidence when the description shown names none of it and the match rests on the rest of the company's profile.

How many familial hypercholesterolemia treatment companies are there worldwide?

RevenueBase identified 61 familial hypercholesterolemia treatment companies worldwide as of September 2026, matched on each company's own description of what it does rather than its industry code.

What is the largest company on this list?

Biomarin Pharmaceutical INC, headquartered in San Rafael, tops this list with the largest team on record.

Which country has the most familial hypercholesterolemia treatment companies?

The United States leads with 34 companies. Every familial hypercholesterolemia treatment companies list by country and city is linked on this page.

Which city has the most familial hypercholesterolemia treatment companies?

Boston leads with 3 headquartered companies, followed by Amsterdam (2) and Cambridge (2).

How do I get contact information for these companies?

A free RevenueBase account lets you run this exact search and work the top results, verified emails and direct dials included. Upgrading unlocks all 2,258 verified decision-maker contacts tracked across these 61 companies.

How current is this list?

The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 19, 2026.

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About this data

RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase Smart Search, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.